1 active not recruiting, 1 available, 1 completed, 1 recruiting. 1 Phase 1, 1 Phase 2, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 1 | Recruiting 1 |
| Phase 2 | 1 | Completed 1 |
| Phase 3 | 1 | Active not recruiting 1 |
Follow Pyruvate Dehydrogenase Complex Deficiency: new trials by email Literature Review of Pyruvate Dehydrogenase Complex Deficiency trials Research Report on Pyruvate Dehydrogenase Complex Deficiency
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency Triheptanoin - Pyruvate Dehydrogenase Complex Deficiency | Phase 1 | Recruiting | Jirair Krikor Bedoyan | 6 | 2024-07-11 | 2026-02-17 |
| Natural History Study of Pyruvate Dehydrogenase Deficiency Pyruvate Dehydrogenase Complex Deficiency, Pyruvate Dehydrogenase E1 Alpha Deficiency | Unknown | Great Ormond Street Hospital for Children NHS Foundation Trust | 50 | 2020-11-01 | 2023-12-05 | |
| Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency: Dichloroacetate (DCA), Placebo, Genotype - Pyruvate Dehydrogenase Complex Deficiency | Phase 3 | Active not recruiting | Saol Therapeutics Inc | 34 | 2020-07-14 | 2025-05-04 |
| Use of Phenylbutyrate Therapy for Patients With Pyruvate Dehydrogenase Complex Deficiency. sodium phenylbutyrate - Pyruvate Dehydrogenase Complex Deficiency | Phase 2 | Completed | Fondazione Telethon | 1 | 2018-10-01 | 2021-10-13 |
| Expanded Access Treatment Protocol With DCA for Patients With PDCD Dichloroacetate (DCA) - Pyruvate Dehydrogenase Complex Deficiency | Available | Saol Therapeutics Inc | 2025-05-08 |
Pyruvate Dehydrogenase Complex Deficiency Disease (5) · Pyruvate Dehydrogenase E1 Alpha Deficiency (1) · Pyruvate Dehydrogenase E1-Beta Deficiency (1) · Pyruvate Dehydrogenase E2 Deficiency (1) · Pyruvate dehydrogenase phosphatase deficiency (1)
Brain Diseases (5) · Brain Diseases, Metabolic (5) · Brain Diseases, Metabolic, Inborn (5) · Carbohydrate Metabolism, Inborn Errors (5) · Central Nervous System Diseases (5) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (5) · Genetic Diseases, Inborn (5) · Genetic Diseases, X-Linked (5) · Heredodegenerative Disorders, Nervous System (5) · Intellectual Disability (5) · Metabolic Diseases (5) · Metabolism, Inborn Errors (5) · Mitochondrial Diseases (5) · Nervous System Diseases (5) · Neurobehavioral Manifestations (5)
Dichloroacetic Acid (2) · 4-phenylbutyric acid (1) · Genotype (1) · triheptanoin (1)
Saol Therapeutics Inc (2) · Fondazione Telethon (1) · Great Ormond Street Hospital for Children NHS Foundation Trust (1) · Jirair Krikor Bedoyan (1)
United States (2) · Italy (1) · United Kingdom (1)
Jirair Bedoyan, MD, PhD (1) · Kiki Diorgu, M.D. (1) · Richard Neibeger, MD (1) · Shamima Rahman, PhD (1)